An investigator-initiated open-label clinical trial of vismodegib as a neoadjuvant to surgery for high-risk basal cell carcinoma - 16/10/14
Abstract |
Background |
Vismodegib is an oral hedgehog-pathway inhibitor approved for advanced basal cell carcinoma (BCC). Although most BCCs are amenable to surgery, excision of large tumors in aesthetically sensitive sites may compromise function or cosmesis.
Objective |
We sought to evaluate the reduction in BCC surgical defect area after 3 to 6 months of neoadjuvant vismodegib.
Methods |
This was an open-label, single-arm intervention trial with a primary outcome of change in target-tumor surgical defect area pre- and post-vismodegib (150 mg/d). Secondary outcomes were change in tumor area and tolerability.
Results |
Eleven of 15 enrolled patients, aged 39 to 100 years, completed the trial. Thirteen target tumors were excised after a mean of 4 ± 2 months of vismodegib. In all, 29% (4 of 14 patients) could not complete more than 3 months because of vismodegib-related side effects. The mean baseline target-tumor diameter was 3.2 cm, and 10 of 13 tumors occurred on the face. Overall, vismodegib reduced the surgical defect area by 27% (95% confidence interval –45.7% to –7.9%; P = .006) from baseline. Vismodegib was not effective in patients who received less than 3 months. Over a mean follow-up of 11.5 (range 4-21) months for all tumors, only 1 tumor recurred at 17 months post-Mohs micrographic surgery.
Limitations |
Short follow-up time and no placebo control are limitations.
Conclusion |
Neoadjuvant vismodegib appears to reduce surgical defect area when taken for 3 months or longer for nonrecurrent BCCs in functionally sensitive locations. Further studies with larger sample sizes and long-term follow-up are warranted.
Le texte complet de cet article est disponible en PDF.Key words : basal cell carcinoma, Mohs, neoadjuvant, surgical defect, surgery, vismodegib
Abbreviations used : BCC, BCNS, CI, NCCN
Plan
| Dr Ally and Dr Aasi contributed equally to this work. |
|
| This investigator-initiated trial was supported in part by a Damon Runyon Cancer Research Foundation Clinical Investigator Award (CI-54-11 to Dr Tang). |
|
| Disclosure: Drs Tang, Chang, and Oro have been investigators in studies sponsored by Genentech. Dr Oro has also been an investigator in studies sponsored by Infinity and Novartis and Dr Chang is also a clinical investigator for studies sponsored by Novartis and Lilly. Drs Ally, Aasi, Wysong, and Kim; Ms Teng; Mr Anderson; and Ms Bailey-Healy have no conflicts of interest to declare. |
Vol 71 - N° 5
P. 904 - novembre 2014 Retour au numéro
Article précédent
- Burden of disease caused by keratinocyte cancer has increased in The Netherlands since 1989
- Loes M. Hollestein, Esther de Vries, Mieke J. Aarts, Caroline Schroten, Tamar E.C. Nijsten
- The role of in vivo confocal microscopy in the diagnosis of eyelid margin tumors: 47 cases
- Elisa Cinotti, Jean Luc Perrot, Nelly Campolmi, Bruno Labeille, Marine Espinasse, Damien Grivet, Gilles Thuret, Philippe Gain, Catherine Douchet, Fabien Forest, Maher Haouas, Frédéric Cambazard
Bienvenue sur EM-consulte, la référence des professionnels de santé.
L’accès au texte intégral de cet article nécessite un abonnement.
Déjà abonné à cette revue ?